CN116194154A - 使用aav载体的plakophilin-2(pkp2)基因疗法 - Google Patents

使用aav载体的plakophilin-2(pkp2)基因疗法 Download PDF

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Publication number
CN116194154A
CN116194154A CN202180058096.5A CN202180058096A CN116194154A CN 116194154 A CN116194154 A CN 116194154A CN 202180058096 A CN202180058096 A CN 202180058096A CN 116194154 A CN116194154 A CN 116194154A
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vector
polynucleotide
ser
gly
leu
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C·D·赫尔佐格
C·B·萨克拉门多
R·普拉巴卡尔
D·里克斯
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Spaceship Seven LLC
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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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CN202180058096.5A 2020-08-07 2021-08-09 使用aav载体的plakophilin-2(pkp2)基因疗法 Pending CN116194154A (zh)

Applications Claiming Priority (3)

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US202063063032P 2020-08-07 2020-08-07
US63/063,032 2020-08-07
PCT/US2021/045220 WO2022032226A1 (en) 2020-08-07 2021-08-09 Plakophilin-2 (pkp2) gene therapy using aav vector

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CN116194154A true CN116194154A (zh) 2023-05-30

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US (2) US20220168447A1 (pt)
EP (1) EP4192487A4 (pt)
JP (1) JP2023538519A (pt)
KR (1) KR20230043869A (pt)
CN (1) CN116194154A (pt)
AU (1) AU2021320902A1 (pt)
BR (1) BR112023001852A2 (pt)
CA (1) CA3185267A1 (pt)
IL (1) IL300263A (pt)
MX (1) MX2023001615A (pt)
WO (1) WO2022032226A1 (pt)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
US11781156B2 (en) * 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2022239846A1 (en) * 2021-03-19 2023-10-26 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
MX2024000175A (es) * 2021-07-08 2024-03-05 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia.
WO2023200742A2 (en) * 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Capsids for plakophillin-2 gene therapy
AR129035A1 (es) * 2022-04-11 2024-07-10 Tenaya Therapeutics Inc Métodos de tratamiento con la terapia génica con placofilina-2
TW202421788A (zh) * 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用ａａｖ基因療法載體治療致心律不整性心肌病
WO2024148332A1 (en) * 2023-01-06 2024-07-11 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
WO2024193638A1 (en) * 2023-03-22 2024-09-26 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of wilson disease

Family Cites Families (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
ATE123059T1 (de) 1987-09-17 1995-06-15 Massachusetts Inst Technology Menschliche, erythroid-spezifische transkriptionsenhancer.
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ATE237694T1 (de) 1991-08-20 2003-05-15 Us Gov Health & Human Serv Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
CA2156725A1 (en) 1993-02-22 1994-09-01 Warren S. Pear Production of high titer helper-free retroviruses by transient transfection
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
EP1792997B1 (en) 1999-10-12 2011-11-02 Institut Pasteur Lentiviral triplex DNA, and vectors and recombinant cells containing lentiviral triplex DNA
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
US20070037165A1 (en) * 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
GB0024550D0 (pt) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
IL160132A0 (en) 2001-08-02 2004-06-20 Inst Clayton De La Rech Methods and compositions relating to improved lentiviral vector production systems
NZ532060A (en) 2001-10-02 2005-11-25 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications
PL222683B1 (pl) 2001-11-13 2016-08-31 Univ Pennsylvania Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), sposoby ich wytwarzania, pakująca komórka gospodarza, kompozycja zawierająca wirus, zastosowanie wirusa, wyizolowane wirusy AAV, białka,sztuczne białka kapsydu, cząsteczki, komórki gospodarza, sposoby dostarczania transgenu, sposób identyfikacji serotypu, zestaw diagnostyczny, sposób izolacji nowych wirusów, rekombinowana komórka
AU2003234337B2 (en) 2002-05-02 2008-08-07 University Of Rochester Vectors having both isoforms of beta-hexosaminidase
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法
JP2006524051A (ja) 2003-04-24 2006-10-26 フォンダッチォーネ・セントロ・サン・ラファエル・デル・モンテ・タボール 両方向性合成プロモーターを備えたレンチウイルスベクターおよびその使用
US7960100B1 (en) * 2004-04-30 2011-06-14 Celera Corporation Colon cancer targets and uses thereof
US20070048285A1 (en) 2005-08-24 2007-03-01 Christopher Baum Self-inactivating retroviral vector
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
AU2008262478B9 (en) 2007-05-29 2014-06-19 Aldevron, L.L.C. Vectors and methods for genetic immunization
CA3002933A1 (en) 2007-07-14 2009-01-22 Beverly L. Davidson Methods and compositions for treating brain diseases
WO2009126894A2 (en) * 2008-04-11 2009-10-15 Massachusetts Eye And Ear Infirmary Methods and compositions for the diagnosis and treatment of angiogenic disorders
US8556882B2 (en) 2009-04-30 2013-10-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
ES2757623T3 (es) 2012-07-25 2020-04-29 Broad Inst Inc Proteínas de unión a ADN inducibles y herramientas de perturbación genómica y aplicaciones de las mismas
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
CN107405411A (zh) 2014-05-01 2017-11-28 华盛顿大学 使用腺病毒载体的体内基因改造
EP3913061A1 (en) 2014-05-02 2021-11-24 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US11030531B2 (en) 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
EP3247808B1 (en) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
KR20180016722A (ko) 2015-03-10 2018-02-19 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
AU2016275909A1 (en) 2015-05-13 2017-11-09 The Trustees Of The University Of Pennsylvania AAV-mediated expression of anti-influenza antibodies and methods of use thereof
JP6976244B2 (ja) * 2015-11-11 2021-12-08 プレシゲン，インコーポレイテッド 心臓状態および他の病態の処置のための複数の生物学的に活性なポリペプチドを単一のベクターから発現させるための組成物および方法
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
PT3405215T (pt) 2016-01-19 2022-09-20 Univ California Métodos para o tratamento da doença de danon e outros transtornos da autofagia
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
KR20240096679A (ko) 2016-04-20 2024-06-26 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
SG11201901718VA (en) 2016-09-08 2019-03-28 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia
WO2018060097A1 (en) 2016-09-30 2018-04-05 Laboratorios Del Dr. Esteve, S. A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
US20180326022A1 (en) * 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
WO2018201065A1 (en) 2017-04-27 2018-11-01 Fred Hutchinson Cancer Research Center Therapeutic formulations containing cd34+ stem cells derived from negative selection
EP3622062A4 (en) 2017-05-10 2020-10-14 The Regents of the University of California DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9
AU2018335401A1 (en) * 2017-09-20 2020-04-02 The Regents Of The University Of California A gene therapy strategy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy
US20200308582A1 (en) * 2017-10-12 2020-10-01 Universität Heidelberg Kcnk3-based gene therapy of cardiac arrhythmia
BR112020007444A2 (pt) 2017-10-16 2020-10-20 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. vetores lentivirais para a liberação de pklr para tratar deficiência de piruvato quinase
MX2020010718A (es) 2018-04-11 2021-01-20 Spacecraft Seven Llc Composiciones y metodos para el trasplante de celulas madre.
JP2021522273A (ja) * 2018-04-27 2021-08-30 ロケット ファーマシューティカルズ リミテッド Ｃｎｓ変性のための遺伝子治療法
JP2021521833A (ja) 2018-04-27 2021-08-30 ウニベルジテート ハイデルベルク 筋疾患の治療のための改変ａａｖカプシドポリペプチド
EP3820537A4 (en) 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
MX2021001292A (es) 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
KR20210043580A (ko) 2018-08-10 2021-04-21 리젠엑스바이오 인크. 재조합 aav 생산을 위한 규모 조정 가능한 방법
BR112021002765A2 (pt) 2018-08-16 2021-07-20 Spacecraft Seven, Llc métodos de produção para vetores virais
US20220088052A1 (en) 2019-01-22 2022-03-24 Koninklijke Nederlandse Akademie Van Wetenschappen Tfap2 inhibition for treating cardiac disease involving fibro-fatty replacement
CA3128514A1 (en) 2019-02-12 2020-08-20 Spacecraft Seven, Llc Gene therapy vectors for treatment of danon disease
CN113994008A (zh) 2019-05-23 2022-01-28 太空飞船七有限责任公司 用于婴儿恶性骨硬化病的基因治疗载体
KR20220066914A (ko) * 2019-09-20 2022-05-24 유씨엘 비즈니스 리미티드 부정맥 유발성 우심실 심근병증의 유전자 요법 조성물 및 치료
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
JPWO2021187380A1 (pt) 2020-03-16 2021-09-23
KR20230004719A (ko) 2020-04-20 2023-01-06 테나야 테라퓨틱스, 인코포레이티드 조작된 캡시드를 갖는 아데노-연관 바이러스
JP2023535121A (ja) 2020-05-20 2023-08-16 スペースクラフト セブン リミテッド ライアビリティ カンパニー 操作されたパーキン及びその使用
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
US20230257431A1 (en) 2020-08-05 2023-08-17 Spacecraft Seven, Llc Csrp3 (cysteine and glycine rich protein 3) gene therapy
US20230272422A1 (en) 2020-08-05 2023-08-31 Spacecraft Seven, Llc Adeno-associated viral vector for glut1 expression and uses thereof
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
EP4090752A4 (en) 2020-10-09 2023-08-30 Tenaya Therapeutics, Inc. METHODS AND COMPOSITIONS FOR PLAKOPHILIN 2-BASED GENE THERAPY
MX2023006694A (es) 2020-12-07 2023-08-25 Spacecraft Seven Llc Tratamiento de la enfermedad de danon.
AU2022239846A1 (en) 2021-03-19 2023-10-26 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
EP4323534A1 (en) 2021-04-12 2024-02-21 The Regents of the University of California Gene therapy for arrhythmogenic right ventricular cardiomyopathy
MX2024000175A (es) 2021-07-08 2024-03-05 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia.

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